Amicus gets hit with an 'unexpected' setback on a gene therapy tapped to lead $600M SPAC spinout
Amicus has taken the axe and chopped a gene therapy for Batten disease out of the pipeline after an “unexpected” setback in the clinic.
In a corporate update on Monday, the biotech reported that its long-term extension data from a Phase I/II study revealed that the initial stabilization they had achieved in patients during the first 2 years wasn’t sustainable. So researchers are dropping the CLN6 Batten disease gene therapy program and turning to a CLN3 program, also for Batten disease, with a “higher dose, different promoter, and intra-cisterna magna route of delivery.”
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January 17, 2022 06:00 AM EST
ZS Perspective: 3 Predictions on the Future of Cell & Gene Therapies
Ben Hohn
The field of cell and gene therapies (C>s) has seen a renaissance, with first generation commercial therapies such as Kymriah, Yescarta, and Luxturna laying the groundwork for an incoming wave of potentially transformative C>s that aim to address diverse disease areas. With this renaissance comes several potential opportunities, of which we discuss three predictions below.
Allogenic Natural Killer (NK) Cells have the potential to displace current Cell Therapies in oncology if proven durable.
Despite being early in development, Allogenic NKs are proving to be an attractive new treatment paradigm in oncology. The question of durability of response with allogenic therapies is still an unknown. Fate Therapeutics’ recent phase 1 data for FT516 showed relatively quicker relapses vs already approved autologous CAR-Ts. However, other manufacturers, like Allogene for their allogenic CAR-T therapy ALLO-501A, are exploring novel lymphodepletion approaches to improve persistence of allogenic cells. Nevertheless, allogenic NKs demonstrate a strong value proposition relative to their T cell counterparts due to comparable response rates (so far) combined with the added advantage of a significantly safer AE profile. Specifically, little to no risk of graft versus host disease (GvHD), cytotoxic release syndrome (CRS), and neurotoxicity (NT) have been seen so far with allogenic NK cells (Fig. 1). In addition, being able to harness an allogenic cell source gives way to operational advantages as “off-the-shelf” products provide improved turnaround time (TAT), scalability, and potentially reduced cost. NKs are currently in development for a variety of overlapping hematological indications with chimeric antigen receptor T cells (CAR-Ts) today, and the question remains to what extent they will disrupt the current cell therapy landscape. Click for more details.
Latest news on Pfizer's $3B+ JAK1 win; Pacts over M&A at #JPM22; 2021 by the numbers; Biogen's Aduhelm reckoning; The story of sotrovimab; and more
Amber Tong
Senior Editor
Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.
For those of you who attended #JPM22 in any shape or form, we hope you had a fruitful time. Regardless of how you spent the past hectic week, may your weekend be just what you need it to be.
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January 14, 2022 04:29 PM ESTUpdated January 14, 07:04 PM
A $3B+ peak sales win? Pfizer thinks so, as FDA offers a tardy green light to its JAK1 drug abrocitinib
John Carroll
Editor & Founder
Back in the fall of 2020, newly crowned Pfizer chief Albert Bourla confidently put their JAK1 inhibitor abrocitinib at the top of the list of blockbuster drugs in the late-stage pipeline with a $3 billion-plus peak sales estimate.
Since then it’s been subjected to serious criticism for the safety warnings associated with the class, held back by a cautious FDA and questioned when researchers rolled out a top-line boast that their heavyweight contender had beaten the champ in the field of atopic dermatitis — Dupixent — in a head-to-head study.
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Rob Califf advances as Biden's FDA nominee, with a close committee vote
Zachary Brennan
Senior Editor
Rob Califf’s second confirmation process as FDA commissioner is already much more difficult than his near unanimous confirmation under the Obama administration.
The Senate Health Committee on Thursday voted 13-8 in favor of advancing Califf’s nomination to a full Senate vote. Several Democrats voted against Califf, including Sen. Bernie Sanders and Sen. Maggie Hassan. Several other Democrats who aren’t on the committee, like West Virginia’s Joe Manchin and Ed Markey of Massachusetts, also said Thursday that they would not vote for Califf. Markey, Hassan and Manchin all previously expressed reservations about the prospect of Janet Woodcock as an FDA commissioner nominee too.
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Michel Vounatsos, Biogen CEO (World Economic Forum/Ciaran McCrickard)
Biogen vows to fight CMS' draft coverage decision for Aduhelm before April finalization
Zachary Brennan
Senior Editor
Biogen executives made clear in an investor call Thursday they are not preparing to run a new CMS-approved clinical trial for their controversial Alzheimer’s drug anytime soon.
As requested in a draft national coverage decision from CMS earlier this week, Biogen and other anti-amyloid drugs will need to show “a meaningful improvement in health outcomes” for Alzheimer’s patients in a randomized, placebo-controlled trial to get paid for their drugs, rather than just the reduction in amyloid plaques that won Aduhelm its accelerated approval in June.
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CRO owner pleads guilty to obstructing FDA investigation into falsified clinical trial data
Zachary Brennan
Senior Editor
The co-owner of a Florida-based clinical research site pleaded guilty to lying to an FDA investigator during a 2017 inspection, revealing that she falsely portrayed part of a GlaxoSmithKline pediatric asthma study as legitimate, when in fact she knew that certain data had been falsified, the Department of Justice said Wednesday.
Three other employees — Yvelice Villaman Bencosme, Lisett Raventos and Maytee Lledo — previously pleaded guilty and were sentenced in connection with falsifying data associated with the trial at the CRO Unlimited Medical Research.
Cancer powerhouse AstraZeneca rolls the dice on a $75M cash bet on a buzzy upstart in the oncology field
John Carroll
Editor & Founder
After establishing itself in the front ranks of cancer drug developers and marketers, AstraZeneca is putting its scientific shoulder — and a significant amount of cash — behind the wheel of a brash new upstart in the biotech world.
The pharma giant trumpeted news this morning that it is handing over $75 million upfront to ally itself with Scorpion Therapeutics, one of those biotechs that was newly birthed by some top scientific, venture and executive talent and bequeathed with a fortune by way of a bankroll to advance an only hazily explained drug platform. And they are still very much in the discovery and preclinical phase.
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‘Skinny labels’ on generics can save patients money, research shows, but recent court decisions cloud future
Zachary Brennan
Senior Editor
New research shows how generic drug companies can successfully market a limited number of approved indications for a brand name drug, prior to coming to market for all of the indications. But several recent court decisions have created a layer of uncertainty around these so-called “skinny” labels.
While courts have generally allowed generic manufacturers to use their statutorily permitted skinny-label approvals, last summer, a federal circuit court found that Teva Pharmaceuticals was liable for inducing prescribers and patients to infringe GlaxoSmithKline’s patents through advertising and marketing practices that suggested Teva’s generic, with its skinny label, could be employed for the patented uses.
A patient in Alaska receiving an antibody infusion to prevent Covid hospitalizations in September. All but one of these treatments has been rendered useless by Omicron (Rick Bowmer/AP Images)
How a tiny Swiss lab and two old blood samples created one of the only effective drugs against Omicron (and why we have so little of it)
Jason Mast
Editor
Exactly a decade before a novel coronavirus broke out in Wuhan, Davide Corti — a newly-minted immunologist with frameless glasses and a quick laugh — walked into a cramped lab on the top floor of an office building two hours outside Zurich. He had only enough money for two technicians and the ceiling was so low in parts that short stature was a job requirement, but Corti believed it’d be enough to test an idea he thought could change medicine.
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